Investigation Links 6-Year-Old's Death to $800,000 Gene Therapy Trial, Exposing Safety Failures
Updated
Updated · Futura · Jul 31
Investigation Links 6-Year-Old's Death to $800,000 Gene Therapy Trial, Exposing Safety Failures
3 articles · Updated · Futura · Jul 31
Summary
A 6-year-old Chinese girl died days after a bespoke CRISPR gene therapy for Snijders Blok-Campeau syndrome, and hospital records reviewed by reporters tied the death to the experimental treatment.
The likely cause was thrombotic microangiopathy — a severe immune reaction to the viral vector — after she developed high fever, kidney failure and a sharp platelet drop following injection into cerebrospinal fluid.
The investigation found key safeguards were missed: macaque studies had shown serious liver and immune toxicity, those findings were apparently not fully shared with the hospital ethics committee, and experts said lower-dose testing should have come first.
The trial was built around Mei alone, with her parents contributing about $800,000, yet public trial registries were not properly updated and a later Nature paper omitted both her treatment and her death.
For gene-therapy researchers, the case underscores broader concerns about uneven oversight and limited transparency in some China-led experimental trials as biotech competition intensifies.